The International LAM Registry: a component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform.

Détails

ID Serval
serval:BIB_92A360AE6E6D
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
The International LAM Registry: a component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform.
Périodique
Lymphatic Research and Biology
Auteur(s)
Nurok M., Eslick I., Carvalho C.R., Costabel U., D'Armiento J., Glanville A.R., Harari S., Henske E.P., Inoue Y., Johnson S.R., Lacronique J., Lazor R., Moss J., Ruoss S.J., Ryu J.H., Seyama K., Watz H., Xu K.F., Hohmann E.L., Moss F.
ISSN
1539-6851
Statut éditorial
Publié
Date de publication
2010
Volume
8
Numéro
1
Pages
81-87
Langue
anglais
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Résumé
BACKGROUND: A relative inability to capture a sufficiently large patient population in any one geographic location has traditionally limited research into rare diseases.
METHODS AND RESULTS: Clinicians interested in the rare disease lymphangioleiomyomatosis (LAM) have worked with the LAM Treatment Alliance, the MIT Media Lab, and Clozure Associates to cooperate in the design of a state-of-the-art data coordination platform that can be used for clinical trials and other research focused on the global LAM patient population. This platform is a component of a set of web-based resources, including a patient self-report data portal, aimed at accelerating research in rare diseases in a rigorous fashion.
CONCLUSIONS: Collaboration between clinicians, researchers, advocacy groups, and patients can create essential community resource infrastructure to accelerate rare disease research. The International LAM Registry is an example of such an effort. 82.
Mots-clé
Clinical Trials as Topic, Computer-Assisted Instruction, Humans, Lymphangioleiomyomatosis/diagnosis, Lymphangioleiomyomatosis/genetics, Rare Diseases/diagnosis, Rare Diseases/genetics, Registries
Pubmed
Web of science
Création de la notice
08/06/2010 16:02
Dernière modification de la notice
20/08/2019 15:55
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